Blue­print Med­i­cines po­ten­tial­ly de­lays Ay­vak­it de­ci­sion; Con­trol beats treat­ment in mesothe­lioma tri­al

Blue­print Med­i­cines filed an amend­ment to its ap­pli­ca­tion to get the gas­troin­testi­nal stro­mal tu­mor (GIST) drug Ay­vak­it ap­proved in fourth-line GIST, the com­pa­ny dis­closed in the prospec­tus for a new $325 mil­lion pub­lic of­fer­ing.  Blue­print got a big ac­cel­er­at­ed OK on the drug this month in a par­tic­u­lar mu­ta­tion, but be­cause the FDA de­cid­ed to split their re­view in two, they didn’t hear on fourth-line GIST. They were sup­posed to hear be­fore Feb­ru­ary 14, but this amend­ment could push that date back by 3 months. Blue­print wrote that the amend­ment is de­signed to al­low the com­pa­ny to com­ply with the FDA’s re­quest for da­ta from the Phase III VOY­AGER be­fore they give a judg­ment.

→ Tar­go­v­ax has re­leased ear­ly da­ta on their ex­per­i­men­tal drug for mesothe­lioma, and they’re not great. The tri­al test­ed ON­COS-102 on 31 pa­tients, in­clud­ing one co­hort of first-line pa­tients and one co­hort of sec­ond-line pa­tients. The re­sponse rate was high­er in the con­trol groups for both first-line and sec­ond-line pa­tients, than in the two treat­ment groups. Tar­go­v­ax said those num­bers may have been skewed by the sam­ple size, and they saw promis­ing clin­i­cal ac­tiv­i­ty – a com­mon re­frain from drug com­pa­nies faced with a failed tri­al, but one that may have some mer­it here: There were on­ly 5 sec­ond-line pa­tients in the con­trol group and they had an eye-pop­ping 60% re­sponse rate from the stan­dard-of-care (as op­posed to 11% in the ON­COS-102 group). Tar­go­v­ax said they will now ex­plore com­bin­ing their ade­n­ovirus-based ther­a­py with a check­point ther­a­py.

Jazz Phar­ma­ceu­ti­cals has sub­mit­ted an NDA for their nar­colep­sy drug, JZP-258, and re­deemed a pri­or­i­ty re­view vouch­er for it. The drug is de­signed to treat day­time sleepi­ness and cat­a­plexy – sud­den mus­cle weak­ness, of­ten in re­sponse to a trig­ger – in pa­tients over the age of 7. The point of the drug is to bring the same ef­fi­ca­cy as Jazz’s long-run­ning nar­colep­sy treat­ment Xyrem but with 1,000 to 1,500 mil­ligrams less sodi­um. Xyrem, or sodi­um oxy­bate, gen­er­al­ly push­es pa­tients’ sodi­um in­take well over the Amer­i­can Heart As­so­ci­a­tion’s rec­om­mend­ed 1,500 mil­ligrams per day, po­ten­tial­ly caus­ing long-term car­dio­vas­cu­lar ef­fects.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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Mickey Kertesz, KidsandArtOrg via YouTube

Soft­Bank's newest, $165M biotech in­vest­ment looks for in­fec­tious traces in the blood

SoftBank has found its newest biotech investment.

The Japanese bank has invested $165 million into Karius, a company that uses blood tests to diagnose infectious diseases, as part of its new Vision Fund 2. The full scope of the new fund has yet to be announced, but the first and newly-beleaguered Vision Fund poured $100 billion into technology companies, including the biotechs Vir Biotechnology and Roivant and the sequencing company 10x Genomics.

Methicillin-resistant Staph aureus (Shutterstock)

FDA grants ‘break­through’ sta­tus to an­tibi­ot­ic al­ter­na­tive as Con­tra­Fect rush­es to join fight against su­per­bug

An experimental drug that promises to be the first anti-infective agent to prove superior to vancomycin — an antibiotic approved in 1958 — has notched the FDA’s “breakthrough” status.

ContraFect said the designation was based on Phase II data in which exebacase was tested against a superbug known as methicillin-resistant Staph aureus, or MRSA. In a subgroup analysis, the clinical responder rate at day 14 was 42.8% higher than that among those treated with standard of care, the company said (p=0.010).

Zhong Nanshan, CGTN via YouTube

Har­vard joins coro­n­avirus fight with $115 mil­lion and a high-pro­file Chi­nese part­ner

For two months, as the novel coronavirus swelled from a few early cases tied to a Wuhan market to a global epidemic, most of the world’s focus and dollars have flowed toward emergency initiatives: building vaccines at a record pace, plucking experimental antivirals out of freezers to see what sticks and immunizing mice for new antibodies.

Now a new and well-funded collaboration between Harvard and a top Chinese research institute will play the long game. In a 5-year, $115 million initiative backed by China Evergrande Group, researchers from the Harvard Medical School, Harvard T.H. Chan School of Public Health and Guangzhou Institute for Respiratory Health will study the virus in an effort to develop therapies against infections by the novel coronavirus, known as SARS–CoV-2, and to prevent new ones.

No­var­tis gets a boost in block­buster mul­ti­ple scle­ro­sis race with Roche

In the first step of what’s likely to be a long and uphill battle for the drugmaker, the FDA has accepted Novartis’s BLA submission for a new multiple sclerosis drug and given it priority review. The PDUFA date for the potential blockbuster drug is in June.

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Juergen Horn

An­i­mal health vet Juer­gen Horn makes new an­ti­body play for pets, rak­ing $15M in Se­ries A haul

Zoetis forked over $85 million in 2017 to acquire Nexvet Biopharma and its pipeline of monoclonal antibodies. Juergen Horn, Nexvet’s former chief product development officer, has now secured $15 million for his own biologic company for animals: Invetx.

Buoyed by emerging advances in gene therapies for humans, scientists have started looking at harnessing the technology for animals setting up companies such as Penn-partnered Scout Bio and George Church-founded Rejuvenate Bio. But akin to Nexvet, Invetx is working on leveraging the time-tested science of monoclonal antibodies to treat chronic diseases that afflict man’s best friend.

As coro­n­avirus out­break reach­es 'tip­ping point,' GSK lends ad­ju­vant tech to Chi­nese part­ner armed with pre­clin­i­cal vac­cine

As the coronavirus originating out of Wuhan spreads to South Korea, Italy and Iran, stoking already intense fears of a pandemic, GlaxoSmithKline has found another pair of trusted hands to place its adjuvant system. China’s Clover Biopharmaceuticals will add the adjuvant to its preclinical, protein-based vaccine candidate against SARS-CoV-2.

Clover, which is based in the inland city of Chengdu, boasts of a platform dubbed Trimer-Tag that produces covalently-trimerized fusion proteins. Its candidate, COVID-19 S-Trimer, resembles the viral spike (S)-protein found in the virus.